ES3058646T3 - A modified mglur6 promoter and methods of use - Google Patents

A modified mglur6 promoter and methods of use

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Publication number
ES3058646T3
ES3058646T3 ES15713278T ES15713278T ES3058646T3 ES 3058646 T3 ES3058646 T3 ES 3058646T3 ES 15713278 T ES15713278 T ES 15713278T ES 15713278 T ES15713278 T ES 15713278T ES 3058646 T3 ES3058646 T3 ES 3058646T3
Authority
ES
Spain
Prior art keywords
seq
mglur6
nucleic acid
gene
intron
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Active
Application number
ES15713278T
Other languages
English (en)
Spanish (es)
Inventor
Zhuo-Hua Pan
Qi Lu
Tushar H Ganjawala
Jrgang Cheng
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Wayne State University
Original Assignee
Wayne State University
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Wayne State University filed Critical Wayne State University
Application granted granted Critical
Publication of ES3058646T3 publication Critical patent/ES3058646T3/es
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A61K38/1787—Receptors; Cell surface antigens; Cell surface determinants for neuromediators, e.g. serotonin receptor, dopamine receptor
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70571—Receptors; Cell surface antigens; Cell surface determinants for neuromediators, e.g. serotonin receptor, dopamine receptor
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/67—General methods for enhancing the expression
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Cell Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Neurology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Saccharide Compounds (AREA)
  • Peptides Or Proteins (AREA)
ES15713278T 2014-03-11 2015-03-11 A modified mglur6 promoter and methods of use Active ES3058646T3 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201461951360P 2014-03-11 2014-03-11
PCT/US2015/019985 WO2015138616A1 (fr) 2014-03-11 2015-03-11 Promoteur mglur6 modifié et procédés d'utilisation

Publications (1)

Publication Number Publication Date
ES3058646T3 true ES3058646T3 (en) 2026-03-12

Family

ID=52780035

Family Applications (1)

Application Number Title Priority Date Filing Date
ES15713278T Active ES3058646T3 (en) 2014-03-11 2015-03-11 A modified mglur6 promoter and methods of use

Country Status (6)

Country Link
US (3) US10307492B2 (fr)
EP (2) EP4678754A3 (fr)
AU (4) AU2015229464B2 (fr)
CA (2) CA2942324C (fr)
ES (1) ES3058646T3 (fr)
WO (1) WO2015138616A1 (fr)

Families Citing this family (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2714007T3 (es) 2007-04-09 2019-05-24 Univ Florida Composiciones de vectores rAAV que tienen proteínas de la cápside modificadas en tirosina y métodos para su uso
US12358954B2 (en) 2012-05-15 2025-07-15 University Of Florida Research Foundation, Incorporated Capsid-modified rAAV vector compositions and methods therefor
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
EP4678754A3 (fr) * 2014-03-11 2026-04-08 Wayne State University Promoteur mglur6 modifié et procédés d'utilisation
US20170319669A1 (en) 2016-05-03 2017-11-09 Wayne State University Method of enhancing viral-mediated gene delivery
EP3504227A1 (fr) 2016-08-29 2019-07-03 Wayne State University Identification de mutations dans des variants de channelopsine ayant une sensibilité améliorée à la lumière et leurs procédés d'utilisation
EP4653537A3 (fr) * 2016-09-02 2026-01-07 Keio University Rhodopsin-gentherapie zur regeneration des sehvermögens oder zur verhinderung der abnahme des sehvermögens
CA3040179A1 (fr) 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Capsides d'aav modifiees et leurs utilisations
JP2020510424A (ja) * 2017-02-28 2020-04-09 アドヴェラム バイオテクノロジーズ， インコーポレイテッド 改変ａａｖカプシドおよびその使用
EP3727468A4 (fr) * 2017-12-19 2021-09-22 Akouos, Inc. Administration d'anticorps thérapeutiques médiée par aav à l'oreille interne
WO2019200016A1 (fr) 2018-04-10 2019-10-17 President And Fellows Of Harvard College Vecteurs aav codant pour clarine-1 ou gjb2 et utilisations associées
CA3112883A1 (fr) * 2018-10-25 2020-04-30 Baxalta Incorporated Systeme triple plasmide aav
KR102934700B1 (ko) * 2019-03-28 2026-03-09 더 제너럴 하스피탈 코포레이션 트랜스진 발현을 위한 조작된 아데노-연관 (aav) 벡터
US11771741B2 (en) 2019-09-13 2023-10-03 Restore Vision Inc. Nucleic acid construct that encodes chimeric rhodopsin
WO2021049634A1 (fr) 2019-09-13 2021-03-18 株式会社レストアビジョン Construction d'acides nucléiques codant pour la rhodopsine chimère
CN114729009A (zh) 2019-10-31 2022-07-08 伯尔尼大学 用于眼部基因递送的aav载体变体
CN115052988A (zh) * 2019-11-18 2022-09-13 伯尔尼大学 用于眼部基因递送的给光型双极细胞特异性启动子
US12129287B2 (en) 2020-09-14 2024-10-29 President And Fellows Of Harvard College Recombinant adeno associated virus encoding clarin-1 and uses thereof
US12071633B2 (en) * 2020-10-13 2024-08-27 Kriya Therapeutics, Inc. Viral vector constructs for delivery of nucleic acids encoding cytokines and uses thereof for treating cancer
IL303317A (en) 2020-12-01 2023-07-01 Akouos Inc ANTI-NATURAL ANTIBODY STRUCTURES AND RELATED METHODS FOR THE TREATMENT OF SYMPTOMS ASSOCIATED WITH VESTIBULAR SWANNOMA
EP4388107A4 (fr) * 2021-08-20 2025-07-02 Massachusetts Eye & Ear Infirmary Nouvelles méthodes d'utilisation du virus adéno-associé 11 (aav11)
EP4665750A1 (fr) * 2023-02-17 2025-12-24 Ray Therapeutics, Inc. Compositions de thérapie génique et méthodes de traitement de maladies de la rétine
WO2025087987A1 (fr) * 2023-10-23 2025-05-01 Sartorius Stedim Cellca Gmbh Plasmides pour titres aav améliorés et rapports complets vides

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US5872A (en) 1848-10-24 Saddle
US5912122A (en) 1993-06-04 1999-06-15 Sibia Neurosciences, Inc. Nucleic acids encoding and method for detecting nucleic acid encoding human metabotropic glutamate receptor subtype mGluR6
US5872005A (en) 1994-11-03 1999-02-16 Cell Genesys Inc. Packaging cell lines for adeno-associated viral vectors
CA2346262A1 (fr) 1998-09-17 2000-03-23 University Of Florida Methodes de traitement de maladies degeneratives de la retine
AU3755900A (en) 1999-03-15 2000-10-04 Chiron Corporation Use of recombinant gene delivery vectors for treating or preventing diseases of the eye
US20070161031A1 (en) * 2005-12-16 2007-07-12 The Board Of Trustees Of The Leland Stanford Junior University Functional arrays for high throughput characterization of gene expression regulatory elements
CN101484005A (zh) 2006-05-04 2009-07-15 韦恩州立大学 通过向体内递送视紫红质核酸恢复视觉响应
EP1891976A1 (fr) 2006-08-23 2008-02-27 Novartis Forschungsstiftung, Zweigniederlassung Friedrich Miescher Institute for Biomedical Research Usage des genes sensibles à la lumière
US9725485B2 (en) * 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
WO2010011404A2 (fr) 2008-05-20 2010-01-28 Eos Neuroscience, Inc. Vecteurs pour la délivrance de protéines photosensibles et procédés d’utilisation
WO2011140279A1 (fr) 2010-05-04 2011-11-10 Wayne State University Ciblage sous-cellulaire faisant intervenir aav de rhodopsines hétérologues dans des cellules ganglionnaires rétiniennes
CN105755044A (zh) * 2011-04-22 2016-07-13 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体及其使用方法
CA2866405A1 (fr) 2012-03-05 2013-09-12 Wayne State University Identification de mutations de channelrhodopsine-2 (chop2) et procedes d'utilisation
EP4678754A3 (fr) * 2014-03-11 2026-04-08 Wayne State University Promoteur mglur6 modifié et procédés d'utilisation

Also Published As

Publication number Publication date
US11583595B2 (en) 2023-02-21
US20230310657A1 (en) 2023-10-05
CA2942324C (fr) 2024-05-14
US20170021038A1 (en) 2017-01-26
AU2023220319A1 (en) 2023-09-14
CA2942324A1 (fr) 2015-09-17
AU2021204416B2 (en) 2023-06-01
EP4678754A2 (fr) 2026-01-14
EP3117002A1 (fr) 2017-01-18
EP4678754A3 (fr) 2026-04-08
AU2023220319B2 (en) 2025-08-21
US20200093938A1 (en) 2020-03-26
AU2025271096A1 (en) 2025-12-18
AU2015229464B2 (en) 2021-07-22
AU2021204416A1 (en) 2021-07-22
AU2015229464A1 (en) 2016-10-27
US10307492B2 (en) 2019-06-04
EP3117002B1 (fr) 2025-12-03
CA3234028A1 (fr) 2015-09-17
WO2015138616A1 (fr) 2015-09-17

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